COUR Pharmaceuticals Reports Positive Phase 2a Results for CNP-104 in Primary Biliary Cholangitis

COUR Pharmaceuticals, a clinical-stage biotechnology company, recently announced encouraging 120-day data from its Phase 2a study on CNP-104, a potential treatment for Primary Biliary Cholangitis (PBC). PBC is a chronic liver disease affecting approximately 130,000 people in the U.S. COUR Pharmaceuticals focuses on creating novel, disease-modifying therapies aimed at inducing antigen-specific tolerance for immune-mediated diseases.

Dr. Paul M. Peloso, the Chief Medical Officer at COUR Pharmaceuticals, emphasized the importance of this development. He highlighted that CNP-104 could become the first disease-modifying treatment for PBC. The study's data showed promising results not only in terms of safety and tolerability but also in various immunological and clinical measurements. Specifically, a reduction in liver stiffness was observed in patients treated with CNP-104 compared to those given a placebo. This reduction, measured via Fibroscan, indicates a potential decrease in fibrosis and suggests that CNP-104 could halt the disease's progression.

Key points from the study include:
1. A statistically significant decrease in liver stiffness in patients treated with CNP-104 compared to those who received a placebo.
2. Patients in the placebo group experienced a more significant decrease in albumin levels compared to those treated with CNP-104.
3. CNP-104 induced a favorable T cell response, evidenced by changes in pathogenic CD4 T cell populations and tolerance-inducing CD8 T cells.
4. A notable reduction in Th17 T cells was observed, with statistically higher response rates in the active treatment groups.
5. CNP-104 was found to be safe and well-tolerated, with all related adverse events being mild and no severe adverse events reported.

The Phase 2a study was a randomized clinical trial involving patients aged 18-75 who had not responded to ursodeoxycholic acid (UDCA) and/or obeticholic acid (OCA). Participants were given either 4 mg/kg or 8 mg/kg of CNP-104 or a placebo on two occasions, one week apart. The study involved 41 participants, with a randomization ratio of approximately 1:1:1 for placebo, 4 mg/kg CNP-104, and 8 mg/kg CNP-104. The report focuses on the primary 120-day study period, while a 20-month long-term safety evaluation is still ongoing.

Dr. Christopher Bowlus, the Principal Investigator for the study and a Professor at UC Davis, underscored the significance of these findings. Despite advancements in the PBC treatment landscape, there remains a pressing need for therapies that address the root cause of the disease. According to Dr. Bowlus, current treatments do not target the underlying mechanisms, unlike CNP-104. Although the data is preliminary and involves a small patient sample, the results are promising and warrant further investigation.

CNP-104 is a biodegradable nanoparticle that encapsulates the E2 component of the mitochondrial pyruvate dehydrogenase complex (PDC), a key autoantigen in PBC. The treatment aims to induce tolerance to pathogenic PDC-E2 T-cells, which drive bile duct inflammation, thereby improving liver health. In January 2022, CNP-104 received Fast Track Designation from the FDA, making it potentially eligible for Accelerated Approval and Priority Review if specific criteria are met.

PBC primarily affects women between the ages of 40 and 60 and is one of the leading causes of liver transplants in women. The disease impairs bile flow and leads to the accumulation of toxic bile acids in the liver, causing fibrosis, cirrhosis, and eventual liver failure. Symptoms like fatigue and pruritus significantly impact patients' quality of life. While the exact cause remains unknown, the presence of antimitochondrial antibodies (AMA) targeting the E2 component of PDC is strongly associated with the disease.

COUR Pharmaceuticals is committed to developing treatments for autoimmune diseases and has a pipeline that includes therapies for Myasthenia Gravis, Type 1 Diabetes, Celiac Disease, and PBC.